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Nilson Family
Our youngest son, Henry, was diagnosed with Duchenne in August 2011 – shortly before his 3rd birthday. It was a fluke test that shocked even the doctors as it was […]
Santhera and ReveraGen Announce Positive and Statistically Highly Significant Topline Results with Vamorolone in Pivotal VISION-DMD Study
June 1, 2021 VISION-DMD 24-week study readout: Update from Santhera Dear U.S. Duchenne community, We are happy to provide an update of the VISION-DMD Phase 2b study conducted by Santhera […]
Munoz Family
In 2013, our oldest son, Joshua, was diagnosed with Duchenne muscular dystrophy. He was 5 years old at the time and we were devastated with the diagnosis. After doing some […]
Foster Family
Our son was diagnosed April 1, 2019. We have spent the past 2 years doing research, completing a clinical trial and being an advocate for our son and Duchenne Muscular […]
Edwards Family
We are the Edwards Family in North Dakota. Our 11 year old son Grant has Duchenne. Diagnosed at age 4. Grant’s younger brother Christian age 8 is unaffected. I carry […]
Cazin Family
We have two sons and a daughter and no family history of Duchenne. In April of 2018, we took our kids to the pediatrician for a well check. We mentioned […]
Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material
Results from the first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus Treated patients achieved mean micro-dystrophin expression levels […]
Don’t Miss CureDuchenne at the Gene Therapy for Muscular Disorders Event
With recent clinical updates from Pfizer, Sarepta and Audenteson their programs in muscular disorders, the timely Gene Therapy for Muscular Disorders arrives as the only meeting dedicated to overcoming the translational […]
Pfizer Letter to the Duchenne Community
Update on Pfizer’s Duchenne gene therapy Phase 3 clinical program In line with our continued efforts to work with the Duchenne community and share information as it becomes available, we […]